- Conditions
- Parkinson's Disease
- Interventions
- CERE-120: AAV2-NTN
- Genetic
- Lead sponsor
- Sangamo Therapeutics
- Industry
- Eligibility
- 35 Years to 75 Years
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2005 – 2007
- U.S. locations
- 2
- States / cities
- San Francisco, California • Chicago, Illinois
Search Results
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Search by query text, NCT ID, condition, intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, or age. Results are retrieved from ClinicalTrials.gov and synchronized into the directory. Search pages remain noindex by default.
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Showing 25–48
of 24
matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
- Conditions
- Sickle Cell Disease, Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0), Beta-Thalassemia, Transfusion Dependent Beta-Thalassaemia
- Interventions
- autologous bone marrow derived CD34+ HSPCs electroporated with BCL11A enhancer targeting Cas9 ribonucleoprotein, Sequencing Assay for Variant rs114518452
- Biological · Device
- Lead sponsor
- Daniel Bauer
- Other
- Eligibility
- 13 Years to 40 Years
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2030
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- Cancer Pain
- Interventions
- NP2
- Biological
- Lead sponsor
- Diamyd Inc
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2013
- U.S. locations
- 5
- States / cities
- Miami, Florida • New Orleans, Louisiana • Ann Arbor, Michigan + 2 more
- Conditions
- Prostate Cancer
- Interventions
- Ad5-yCD/mutTKSR39rep-hIL12
- Biological
- Lead sponsor
- Henry Ford Health System
- Other
- Eligibility
- 18 Years and older · Male only
- Enrollment
- 15 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2024
- U.S. locations
- 1
- States / cities
- Detroit, Michigan
- Conditions
- HIV Infections
- Interventions
- WT-gag-TCR modified T cells, α/6-gag-TCR modified T cells, STI or Drug Holiday
- Biological · Other
- Lead sponsor
- University of Pennsylvania
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 2 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2014
- U.S. locations
- 1
- States / cities
- Philadelphia, Pennsylvania
- Conditions
- Parkinson's Disease
- Interventions
- AAV-GAD
- Genetic
- Lead sponsor
- MeiraGTx, LLC
- Industry
- Eligibility
- 25 Years to 86 Years
- Enrollment
- 13 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2029
- U.S. locations
- 6
- States / cities
- New Haven, Connecticut • West Bloomfield, Michigan • New York, New York + 2 more
- Conditions
- MPS IIIA, Sanfilippo Syndrome, Sanfilippo A, Mucopolysaccharidosis III
- Interventions
- UX111, Prophylactic Immunomodulatory (IM) Therapy, Optimized Prophylactic IM Therapy, Adjuvant IM Therapy
- Biological · Drug
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Industry
- Eligibility
- Not listed
- Enrollment
- 36 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2029
- U.S. locations
- 2
- States / cities
- Columbus, Ohio • Pittsburgh, Pennsylvania
- Conditions
- HLA-A*0201 Positive Cells Present, Locally Advanced Malignant Neoplasm, NY-ESO-1 Positive, Unresectable Malignant Neoplasm, Sarcoma
- Interventions
- 18F-FHBG, Aldesleukin, Busulfan, Cellular Therapy, Computed Tomography, Filgrastim, Fludarabine, Leukapheresis, Plerixafor, Positron Emission Tomography
- Other · Biological · Drug + 1 more
- Lead sponsor
- Jonsson Comprehensive Cancer Center
- Other
- Eligibility
- 16 Years and older
- Enrollment
- 5 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2023
- U.S. locations
- 1
- States / cities
- Los Angeles, California
- Conditions
- Glioblastoma Multiforme, Astrocytoma, Grade III
- Interventions
- ADV/HSV-tk (gene therapy)
- Drug
- Lead sponsor
- David Baskin MD
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 62 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2026
- U.S. locations
- 1
- States / cities
- Houston, Texas
- Conditions
- Mesial Temporal Lobe Epilepsy
- Interventions
- AAV9-hSyn1-miGRIK2
- Genetic
- Lead sponsor
- UniQure Biopharma B.V.
- Industry
- Eligibility
- 18 Years to 75 Years
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2031
- U.S. locations
- 18
- States / cities
- Birmingham, Alabama • Phoenix, Arizona • Palo Alto, California + 15 more
- Conditions
- Canavan Disease
- Interventions
- rAAV9-CB6-AspA
- Drug
- Lead sponsor
- University of Florida
- Other
- Eligibility
- 18 Months to 24 Months · Male only
- U.S. locations
- 1
- States / cities
- Gainesville, Florida
- Conditions
- Mucopolysaccharidosis Type II (MPS II)
- Interventions
- RGX-121
- Genetic
- Lead sponsor
- REGENXBIO Inc.
- Industry
- Eligibility
- 5 Years to 17 Years · Male only
- Enrollment
- 6 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2024
- U.S. locations
- 1
- States / cities
- Oakland, California
- Conditions
- Severe Combined Immunodeficiency Syndrome
- Interventions
- CD34+ cells transduced with ADA retrovir
- Drug
- Lead sponsor
- National Human Genome Research Institute (NHGRI)
- NIH
- Eligibility
- 1 Month and older
- Enrollment
- 8 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2001 – 2014
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Hemophilia B
- Interventions
- SB-FIX
- Biological
- Lead sponsor
- Sangamo Therapeutics
- Industry
- Eligibility
- 18 Years and older · Male only
- Enrollment
- 1 participant
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2021
- U.S. locations
- 1
- States / cities
- Washington D.C., District of Columbia
- Conditions
- SMARD1, CMT2S
- Interventions
- Gene Therapy
- Biological
- Lead sponsor
- Megan Waldrop
- Other
- Eligibility
- 2 Months to 14 Years
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2030
- U.S. locations
- 1
- States / cities
- Columbus, Ohio
- Conditions
- Cystic Fibrosis
- Interventions
- SP-101 and doxorubicin Cohort 1, SP-101 and doxorubicin Cohort 2
- Combination Product
- Lead sponsor
- Spirovant Sciences, Inc.
- Industry
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 15 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2026
- U.S. locations
- 4
- States / cities
- Kansas City, Kansas • Boston, Massachusetts • New York, New York + 1 more
- Conditions
- Friedreich Ataxia, Cardiomyopathy, Secondary
- Interventions
- Low dose LX2006, Mid Dose LX2006, High Dose LX2006
- Genetic
- Lead sponsor
- Lexeo Therapeutics
- Industry
- Eligibility
- 18 Years to 50 Years
- Enrollment
- 8 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2029
- U.S. locations
- 3
- States / cities
- Los Angeles, California • Tampa, Florida • Rochester, Minnesota
- Conditions
- Hemophilia A
- Interventions
- SPK-8011
- Genetic
- Lead sponsor
- Spark Therapeutics, Inc.
- Industry
- Eligibility
- 18 Years and older · Male only
- Enrollment
- 25 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2023
- U.S. locations
- 11
- States / cities
- Sacramento, California • Gainesville, Florida • Boston, Massachusetts + 7 more
- Conditions
- Colorectal Cancer, Metastatic Cancer
- Interventions
- NV1020
- Biological
- Lead sponsor
- Memorial Sloan Kettering Cancer Center
- Other
- Eligibility
- 18 Years and older
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2000 – 2009
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Batten Disease, Late-Infantile Neuronal Ceroid Lipofuscinosis
- Interventions
- AAVrh.10CUhCLN2 vector 9.0x10^11 genome copies, AAVrh.10CUhCLN2 vector 2.85x10^11 genome copies
- Biological
- Lead sponsor
- Weill Medical College of Cornell University
- Other
- Eligibility
- 2 Years to 18 Years
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2020
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- AIDS-Related Burkitt Lymphoma, AIDS-Related Diffuse Large B-cell Lymphoma, AIDS-Related Plasmablastic Lymphoma, AIDS-Related Primary Effusion Lymphoma, HIV Infection, AIDS Related Non-Hodgkin Lymphoma
- Interventions
- Prednisone, Rituximab, Etoposide, Doxorubicin Hydrochloride, Vincristine Sulfate, Cyclophosphamide, Filgrastim, Lentivirus Vector rHIV7-shI-TAR-CCR5RZ-transduced Hematopoietic Stem/Progenitor Cells
- Drug · Biological
- Lead sponsor
- City of Hope Medical Center
- Other
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2026
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Intermittent Claudication, Peripheral Vascular Disease, Atherosclerosis
- Interventions
- Ad2/HIF-1α/VP16, Saline (Placebo Control)
- Biological · Other
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- 40 Years to 80 Years
- Enrollment
- 289 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2005 – 2010
- U.S. locations
- 35
- States / cities
- Birmingham, Alabama • Palo Alto, California • Sacramento, California + 28 more
- Conditions
- Muscular Dystrophies, Limb-Girdle
- Interventions
- SRP-9005, Corticosteroid
- Biological · Drug
- Lead sponsor
- Sarepta Therapeutics, Inc.
- Industry
- Eligibility
- 4 Years and older
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2032
- U.S. locations
- 1
- States / cities
- Norfolk, Virginia
- Conditions
- Hemophilia B
- Interventions
- PF-06838435 (formerly SPK-9001)
- Biological
- Lead sponsor
- Pfizer
- Industry
- Eligibility
- 18 Years to 65 Years · Male only
- Enrollment
- 21 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2029
- U.S. locations
- 17
- States / cities
- Sacramento, California • Metairie, Louisiana • New Orleans, Louisiana + 4 more